A Phase 1/2A, Open Label Dose Escalation and Cohort Expansion Study of Orally Administered CA-4948 (IRAK4i) as a Monotherapy in Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Acute Myelogenous Leukemia (AML) or higher-risk Myelodysplastic Syndrome (MDS).

Phase 1: Safety measured by adverse events (AEs), dose-limiting toxicities (DLTs) electrocardiograms (ECGs), chemistry and hematology laboratory values, vital signs, and physical examinations, Phase 2a: Clinical response in AML or hrMDS assessed as follows: • AML: Proportion of patients who achieve CR + CRh • hrMDS: ORR (CR + PR)

Phase 1: PK parameters of CA 4948 measured by maximum plasma concentration (Cmax), trough plasma concentration (Cmin), time to Cmax (Tmax,) area under the concentration-time curve from 0-24 hours (AUC 0-24), area under the concentration-time curve from 0 to infinity (AUC 0-inf), and half-life (T ½), Phase 1: Clinical response in AML :- Proportion of patients who achieve complete response (CR) + complete response with partial hematological recovery (CRh) - Proportion of patients who achieve complete response with incomplete hematological recovery (CRi), or CR, or CRh, or partial response (PR), or morphologic leukemia-free state (MLFS) - Duration of response (DOR) - Time to response, Phase 1: Clinical response in hrMDS: - Overall response rate (ORR): proportion of patients who achieve CR or PR - DOR - Time to response, Phase I: Transfusion independence in AML or hrMDS, Phase 2a: Safety measured by AEs, ECGs, chemistry and hematology laboratory values, vital signs, and physical examinations, Phase 2a: Clinical response in AML or hrMDS assessed as follows:- Proportion of patients with AML who achieve CR, or CRh, or CRi - Proportion of patients with hrMDS who achieve CR, or PR, or mCR, with or without hematological improvement - DOR - Time to responses - Transfusion independence - Overall survival (OS)

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